TRIAC trial in MCT8 patients
Research type
Research Study
Full title
Thyroid hormone analogue therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The TRIAC Trial
IRAS ID
199815
Contact name
Carla Moran
Contact email
Sponsor organisation
Erasmus MC
Eudract number
2014-000178-20
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 0 months, 31 days
Research summary
Thyroid Hormone (TH) is crucial for normal brain development. The TH transporter MCT8, located in the plasma membrane, is crucial for cellular transport of TH. Male patients with a mutation in the X-linked MCT8 gene suffer from severe neurological abnormalities, also known as Allan-Herndon-Dudley syndrome (AHDS). They also have abnormal serum TH levels, i.e. low T4 and high T3 levels. The neurological phenotype entails diminished TH transport into the brain. The elevated serum T3 levels exert deleterious effects on tissues where MCT8 is not important for T3 entry. As a consequence, AHDS patients have a low body weight and muscle mass. At present, no effective treatment is available. Effective therapy should at least restore cellular TH signaling in brain and normalise serum TH levels in AHDS patients. Preliminary results indicate that the T3 analogue, TRIAC (TA3, Tiratricol, Teatrois) fulfils these criteria. We hypothesize that treatment of AHDS patients with TRIAC will improve neurodevelopment and relieve symptoms resulting from toxic T3 levels in peripheral tissues.
REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
16/WM/0294
Date of REC Opinion
5 Aug 2016
REC opinion
Favourable Opinion